Drugs / NTLA-2001
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | UK (MHRA) | — | ATTRV122I amyloidosis | 2020-08-06 | “Submitted first regulatory application to the U.K.’s MHRA to initiate a Phase 1 study of NTLA-2001 for the treatment of transthyretin amyloidosis” intelliatx.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT04601051 | Nov 2020 → Sep 2025 | ATTRV122I amyloidosis, wild type ATTR amyloidosis | Intellia Therapeutics | Completed | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT06128629 | Dec 2023 → Dec 2027 expected | — | Intellia Therapeutics | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 26
| Date | Issuer | Release |
|---|---|---|
| 2026-03-02 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Lift of Clinical Hold on MAGNITUDE Phase 3 Clinical Trial in ATTR-CM intelliatx.com ↗ |
| 2026-01-27 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Lift of Clinical Hold on MAGNITUDE-2 Phase 3 Clinical Trial in ATTRv-PN intelliatx.com ↗ |
| 2025-11-10 | Intellia Therapeutics | Results Intellia Therapeutics Presents Positive Longer-Term Phase 1 Data of Nexiguran Ziclumeran (nex-z) in Patients with Transthyretin (ATTR) Amyloidosis with Cardiomyopathy intelliatx.com ↗ |
| 2025-10-27 | Intellia Therapeutics | Results Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (nex-z) intelliatx.com ↗ |
| 2025-09-25 | Intellia Therapeutics | Results Intellia Therapeutics Announces Positive Longer-Term Phase 1 Data for Nexiguran Ziclumeran (nex-z) in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy intelliatx.com ↗ |
| 2025-05-18 | Intellia Therapeutics | Results Intellia Announces Positive Two-Year Follow-Up Data from Ongoing Phase 1 Study of Nexiguran Ziclumeran (nex-z), in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy at Peripheral Nerve Society Annual Meeting intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). |
| 2025-03-26 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Transthyretin (ATTR) Amyloidosis with Cardiomyopathy intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). |
| 2024-11-25 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy intelliatx.com ↗
It was granted following the FDA’s review of our compelling interim Phase 1 data that indicated our one-time treatment led to rapid, deep and durable TTR reduction, which is expected to halt and potentially reverse the disease. |
| 2024-11-16 | Intellia Therapeutics | Results Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran Ziclumeran (nex-z), an In Vivo CRISPR/Cas9-Based Gene Editing Therapy, May Favorably Impact Disease Progression in Transthyretin (ATTR) Amyloidosis intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. |
| 2024-06-25 | Intellia Therapeutics | Results Intellia Announces Positive Clinical Proof-of-Concept Data for Redosing a CRISPR-Based Therapy with its Proprietary LNP-Based Delivery Platform intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today presented new data demonstrating for the first time the potential for redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy. |
| 2023-11-02 | Intellia Therapeutics | Results Intellia Presents New Interim Data from the Ongoing Phase 1 Study of NTLA-2001 at the 4th International ATTR Amyloidosis Meeting intelliatx.com ↗ |
| 2023-10-18 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Pivotal Phase 3 Trial of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis with Cardiomyopathy intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy. |
| 2022-11-05 | Intellia Therapeutics | Results Intellia Presents Updated Interim Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis at the American Heart Association Scientific Sessions 2022 intelliatx.com ↗
Data presented in late-breaking oral presentation demonstrated deep and consistent TTR reduction following a single dose of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) |
| 2022-09-16 | Intellia Therapeutics | Results Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis. |
| 2022-09-16 | Regeneron Pharmaceuticals | Results Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis regeneron.com ↗ |
| 2022-06-24 | Intellia Therapeutics | Results Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose intelliatx.com ↗
The presentation today included extended follow-up data from 15 patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) treated across four single-ascending dose cohorts in Part 1 of the study. |
| 2022-06-24 | Regeneron Pharmaceuticals | Results Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose regeneron.com ↗
Intellia Therapeutics, Inc. (NASDAQ: NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced additional positive interim data from an ongoing Phase 1 study of their lead investigational in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. |
| 2022-02-28 | Intellia Therapeutics | Results Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein intelliatx.com ↗ |
| 2022-02-28 | Regeneron Pharmaceuticals | Results Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein regeneron.com ↗
Mean serum TTR reduction remained durable through the observation period, with patient follow-up ranging from two to 12 months. |
| 2021-11-22 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces Expansion of Ongoing Phase 1 Study of NTLA-2001 to Include Adults with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM) intelliatx.com ↗ |
| 2021-10-21 | Intellia Therapeutics | Regulatory Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. |
| 2021-06-26 | Intellia Therapeutics | Results Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis intelliatx.com ↗ |
| 2021-06-26 | Regeneron Pharmaceuticals | Results Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis regeneron.com ↗ |
| 2021-03-30 | Intellia Therapeutics | Regulatory Intellia Therapeutics’ Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR Amyloidosis intelliatx.com ↗
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced today that the European Commission (EC) has granted orphan drug designation to NTLA-2001. |
| 2020-10-19 | Intellia Therapeutics | Regulatory Intellia Therapeutics Receives Authorization to Initiate Phase 1 Clinical Trial of NTLA-2001 for Transthyretin Amyloidosis (ATTR) intelliatx.com ↗ |
| 2020-08-06 | Intellia Therapeutics | Regulatory Intellia Therapeutics Announces Second Quarter 2020 Financial Results intelliatx.com ↗
We are very pleased to announce our first regulatory submission to initiate a Phase 1 study of NTLA-2001, which is an important milestone as we deliver on our mission to develop curative, CRISPR/Cas9-based treatments for severe diseases. |
All press releases naming this drug 32 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | NTLA-2001 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04601051 ↗ |
| Modality | CRISPR / gene editing | “It is based on the clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9) system” PMID 34215024 ↗ Jun 2021 |
| Route | Intravenous | “single intravenous infusion” PMID 39555828 ↗ Nov 2024 |
| Target | TTR | “a single guide RNA targeting TTR” PMID 34215024 ↗ Jun 2021 |